Apolipoprotein C-III Inhibition - Killing Two Birds with One Stone?

Kraaijenhof, Jordan M; Stroes, Erik S G · NEJM Evid · 2023

rct · Level II

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Abstract

In this issue of <i>NEJM Evidence</i>, Gaudet et al.<sup>1</sup> report on safety and efficacy of a strategy targeting apolipoprotein C-III (APOC3) using a silencing ribonucleic acid (RNA; ARO-APOC3). In a phase I trial comprising 52 healthy volunteers (triglycerides [TG], >80 mg/dl), 40 patients with hypertriglyceridemia (TG, >300 mg/dl), and 20 patients with chylomicronemia (TG, >880 mg/dl), ARO-APOC3 or placebo was administered subcutaneously either once or twice, with doses given on days 1 and 29. The primary goal was to examine the safety of ARO-APOC3. Injection site reactions were the most commonly observed adverse clinical event; no adverse effects led to discontinuation of the study drug.

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